Medical Devices for Rare Diseases No Longer Need to Use Local IRBs
[Posted on: Thursday, June 8, 2017]
The Humanitarian Use Devices (HUD) form a small group of FDA regulated devices intended to treat rare disease. It is the device equivalent of the orphan drug designation for drug and biologics but much less popular than the orphan drug. There are only 63 HUD devices in the market; each year FDA approved 2-6 of such devices. In the 21st Century Cures Act, an attempt was made to expand the HUD. For a device to be eligible for HUD, it had to have less than 4000 patients per year; the limit was expanded to 8000. Another issue with HUDs is that they could only be used with an IRB approval. Every time an HUD is used, the surgeon needs to get IRB approval. This is because HUDs are approved based on the expected safety profile not the efficacy so by law IRB confirms that the device will be reasonably expected to the safe for the patient it is intended. By a strange quirk of the law, HUDs always were to be regulated by the local IRB, thereby greatly limiting their use since not all clinics where they are implanted have local IRBs. Plus local IRBs may not be as efficient to review and promptly approve the use like the commercial centralized IRBs. Central IRBs are allowed for all other device approvals such as for clinical trials with investigational medical devices. The 21st Century Cures Act, remedied this issue as well to allow HUDs to be regulated by any IRB and hence now they are similar to practically all other products in terms of IRB review. Both the changes in the law became effective this week. While this event affects very few products, its impact on the rare diseases where these devices are used will certainly be significant.